hC Bioscience

Overview
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RNA Therapeutics?
Product stageSegments
Ideation
?
Other RNA-based therapeutics
?

hC Bioscience is a biopharmaceutical company developing first-in-class tRNA-based therapeutics to address protein dysfunction in genetic diseases and cancer. The company's innovative approach uses engineered transfer RNAs (tRNAs) to edit proteins without altering genes, focusing on overwriting nonsense mutations that cause premature termination codons (PTCs). hC Bioscience is advancing two complementary platforms: PTCX ("Patch"), which restores protein function by suppressing PTCs, and SWTX ("Switch"), which targets diseases caused by missense mutations. This gene-agnostic technology has the potential to treat 10-15% of all human diseases caused by genetic errors.

The company's lead candidate, HCB-101, is an anticodon engineered tRNA designed to treat severe hemophilia A. Delivered via lipid nanoparticles to target the liver, HCB-101 aims to restore production of full-length, functional Factor VIII protein in approximately 20% of severe hemophilia A cases caused by PTCs. preclinical data presented in April 2024 demonstrated successful liver targeting in mice and in vitro production of full-length Factor VIII despite the presence of PTCs. hC Bioscience plans to initiate a Phase 1 clinical trial for severe hemophilia A in 2025, with IND-enabling studies underway.


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Disclaimer: This company profile has been generated using data obtained through automated web searches and advanced generative AI technology. While we strive to ensure the accuracy and reliability of our sources, auto-generated information could be outdated or inaccurate and should be verified independently.
HQ location:
One Broadway Cambridge MA USA
Founded year:
2021
Employees:
1-10
IPO status:
Private
Total funding:
USD 40.0 mn
Last Funding:
USD 16.0 mn (Series A; Nov 2022)
Last valuation:
-
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